NIJMEGEN, the Netherlands – 25 October 2022: Khondrion, a clinical-stage biopharmaceutical company discovering and developing therapies targeting primary mitochondrial diseases, today announces that Prof. Dr Jan Smeitink, Chief Executive Officer and Dr Herma Renkema, Chief Scientific Officer, at Khondrion will participate in Targeting Mitochondria 2022, the congress of the World Mitochondria Society (WMS), taking place in Berlin, Germany, from 26- 28 October 2022.
Targeting Mitochondria gathers scientists, physicians and companies working on the development and commercialisation of mitochondria-targeted therapies to discuss the latest progress in mitochondrial disease research.
During Session 6, “WMS Challenges in the Acceleration of Mitochondrial Medicine”, on Friday 28 October, Dr Smeitink will provide a development overview of Khondrion’s lead clinical asset, sonlicromanol, which is currently in development to treat a range of mitochondrial diseases in children and adults.
Prof. Dr Jan Smeitink, Chief Executive Officer of Khondrion, commented: “Opportunities to share knowledge at events, such as Targeting Mitochondria, are vital to progress research and development of mitochondria-targeted therapies and new approaches to tackle these severe and debilitating diseases.”
Sonlicromanol is being developed to treat a range of mitochondrial diseases in children and adults. As a first-in-class, oral small molecule, it targets key underlying mechanisms of mitochondrial disease based on its unique triple mode of action. Sonlicromanol is a reductive and oxidative distress modulator with anti-inflammatory properties thereby modulating redox to help restore the cell’s metabolism, a radical trapping agent preventing lipid peroxidation induced ferroptotic cell death, and an mPGES-1 inhibitor ensuing in anti-inflammatory results. Headline results from the Company’s Phase IIb programme, in adult patients with MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders, are expected later this year.
For more information on, or to register for, Targeting Mitochondria, visit https://wms-site.com/
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Prof. Dr. Jan Smeitink, CEO
Tel: +31 24 7635000
Consilium Strategic Communications
David Daley, Melissa Gardiner, Kris Lam
Tel: +44 20 3709 5700
Khondrion is a clinical stage biopharmaceutical company developing therapies for patients with inherited mitochondrial diseases. Based on proprietary science and a deep biological understanding of mitochondrial dysfunction, the company is advancing its lead drug candidate sonlicromanol, a first-in-class, oral small molecule targeting key underlying mechanisms of mitochondrial disease based on the drug’s unique triple mode of action. Sonlicromanol is a reductive and oxidative distress modulator with anti-inflammatory properties.
One of the most advanced disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol is currently being tested in a Phase IIb trial and a 12-month open-label extension study in adult patients with MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders, as well as in a 6-month Phase II study in children with genetically confirmed primary mitochondrial diseases and who suffer from motor symptoms. The compound has been granted orphan drug designations for the treatment of MELAS, Leigh disease and patients with maternally inherited diabetes and deafness (MIDD) in Europe, and for all inherited mitochondrial respiratory chain disorders in the US. It has also been granted a Rare Pediatric Disease designation in the US for the treatment of MELAS. Sonlicromanol and other compounds from Khondrion’s proprietary library have the potential to be developed for a wide range of diseases and conditions with the aim of benefiting patients whose daily lives are severely impacted by mitochondrial impairment.
For more information visit www.khondrion.com
About mitochondrial disease
Mitochondrial disease occurs when mitochondria, found within all cells of the human body and responsible for producing the energy necessary for cells to function, are defective. This can result in a wide range of serious and debilitating illnesses occurring shortly after birth or later in life. Signs and symptoms of these can include cognitive problems, learning disabilities, blindness, deafness, heart failure, diabetes, fatigue, intolerance to exercise, muscle weakness and gait problems, and stunted growth. Orphan diseases of the oxidative phosphorylation system like Leigh disease, MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders including MIDD (maternally inherited diabetes and deafness), LHON (Leber’s hereditary optic neuropathy) and other respiratory chain/ oxidative phosphorylation disorders, are all examples of mitochondrial disease.